Therapeutic Innovation & Regulatory Science
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Published By Sage Publications

2168-4804, 2168-4790

Author(s):  
Mohammad Reza Miri ◽  
Afshin Zare ◽  
Jamileh Saberzadeh ◽  
Neda Baghban ◽  
Iraj Nabipour ◽  
...  
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Author(s):  
Daniel Tobias Michaeli ◽  
Hasan Basri Yagmur ◽  
Timur Achmadeev ◽  
Thomas Michaeli

Abstract Objectives This study evaluates the association of Biopharma company valuation with the lead drug’s development stage, orphan status, number of indications, and disease area. We also estimated annual returns Bioentrepreneurs and investors can expect from founding and investing in drug development ventures. Methods SDC Thomson Reuter and S&P Capital IQ were screened for majority acquisitions of US and EU Biopharma companies developing new molecular entities for prescription use (SIC code: 2834). Acquisition data were complemented with drug characteristics extracted from clinicaltrials.gov, the US Food and Drug Administration (FDA), and deal announcements. Thereafter, company valuations were combined with previously published clinical development periods alongside orphan-, indication-, and disease-specific success rates to estimate annual returns for investments in drug developing companies. Results Based on a sample of 311 Biopharma acquisitions from 2005 to 2020, companies developing orphan, multi-indication, and oncology drugs were valued significantly higher than their peers during later development stages (p < 0.05). We also estimated significantly higher returns for shareholders of companies with orphan relative to non-orphan-designated lead drugs from Phase 1 to FDA approval (46% vs. 12%, p < 0.001). Drugs developed across multiple indications also provided higher returns than single-indication agents from Pre-Clinic to FDA approval (21% vs. 11%, p < 0.001). Returns for oncology drugs exceeded other disease areas (26% vs. 8%, p < 0.001). Conclusions Clinical and economic conditions surrounding orphan-designated drugs translate to a favorable financial risk-return profile for Bioentrepreneurs and investors. Bioentrepreneurs must be aware of the upside real option value their multi-indication drug could offer when negotiating acquisition or licensing agreements.


Author(s):  
Margaret Gamalo ◽  
Christina Bucci-Rechtweg ◽  
Robert M. Nelson ◽  
Linh Vanh ◽  
Ariel Porcalla ◽  
...  

Author(s):  
Ya Grace Gao ◽  
Samantha Roberts ◽  
Allison Guy

AbstractTo promote the efficient review of oncology drug applications, the US Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) launched the Real-Time Oncology Review (RTOR) pilot program in 2018. RTOR allows FDA to review individual sections of eCTD modules of a drug application for oncology drugs in contrast to requiring the applicant to submit complete modules or the complete application before review is initiated. Initially, the program accepted only supplemental applications with simple study designs and easily interpretable endpoints, but the scope has since been expanded to include applications for New Molecular Entities (NME), and other applications with more complex features. Though many applicants experience faster approvals under RTOR, it is difficult to isolate the effect of the RTOR program on review timelines as its contribution is masked by other expedited programs like priority review and breakthrough therapy designation (BTD). This article discusses the expanded scope of RTOR, its interplay with other OCE initiatives to modernize regulatory review, summarizes Genentech’s experiences in planning RTOR submissions from February 2019 to July 2021, and provides considerations for the future of the program.


Author(s):  
Reema Shah ◽  
Nil Patel ◽  
Yasha Patel ◽  
Michael Toscani ◽  
Joseph Barone ◽  
...  

Abstract Background Melanoma is a skin cancer with a rising worldwide incidence of just over 280,000 individuals with the greatest burden of illness in European, New Zealander, and Australian populations. Patients are diagnosed with melanoma with the mean and median ages being 65 and 59 years old, respectively. Phase 3 trials not only provide a wide representation of the target population but also study the efficacy for a certain intervention. Objective The objective of this literature review is to analyze patient demographics of phase 3 trials for melanoma and identify if there is a true disparity between the clinical trial age demographics and the natural epidemiological age demographics. Data Sources The authors conducted a search on clinicaltrials.gov, a publicly available resource that lists clinical trials and their data. The reported mean and median ages for each trial were extracted after determining if each trial meets our inclusion criteria. Weighted mean and median ages were calculated using an online calculator. Data Summary Data from 35 trials were evaluated with 30 trials reporting a weighted mean age of 55.85 years and 5 trials reporting a weighted median age of 55.14 years. Conclusion Based on the results, melanoma clinical trials enroll patients who are younger than the epidemiological mean and median ages. Due to this underrepresentation of the elderly patients with melanoma, clinical trials may provide limited application for the use of their results.


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